Intraventricular Baclofen for Treatment of Severe Dystonia Associated with Glutaryl‐CoA Dehydrogenase Deficiency (GA1): Report of Two Cases. Issue 3 (5th January 2016)

• Record Type: Journal Article
• Authors: Ghatan, Saadi; Kokoszka, Malgosia A.; Ranney, Anne M.; Strauss, Kevin A.
• Is Part Of: Movement disorders clinical practice. Volume 3:Issue 3(2016:Sep.)
• Page Start: 296
• Page End: 299
• Publication Date: 2016-01-05
• Subjects: secondary dystonia -- DBS -- surgery; Movement Disorders; Movement disorders -- Periodicals; Movement disorders; Periodicals; Periodicals; 616
• DOI: 10.1002/mdc3.12278
• Languages: English
• ISSNs: 2330-1619

Abstract

Abstract: Two individuals with intractable generalized dystonia secondary to glutaric aciduria type 1 (GA1) were treated with continuous intraventricular baclofen (IVB) infusion. On IVB of 220 μg/day, one 10‐year‐old girl had an 85% reduction in dystonia, from Barry‐Albright Dystonia Scale (BADS) score 30.7 to 4.5 (maximum score: 32) at 30 postoperative months. Her enteral dystonia medications were reduced >60%, and she discontinued medications for pain, anxiety, and depression. A second GA1 patient, age 23, experienced a more modest 18% reduction in dystonia (BADS decrease from 29.7 to 24.3) on IVB of 1, 665 μg/day at 14 postoperative months. He substantially reduced his enteral dystonia medications and reported meaningful pain relief. These cases demonstrate that IVB may be a palliative option in the intractable dystonia of GA1. Our provisional observations suggest that IVB may be more beneficial in younger GA1 patients.